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Updated: Dec 4, 2025

A Scalable, Cell-Based Method for the Functional Assessment of Ube3a Variants
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アンジェルマン症候群に対するCas9遺伝子治療は,Ube3a-ATS長い非コーディングRNAを捕らえます

Justin M Wolter1,2,3, Hanqian Mao1,2,3, Giulia Fragola1

  • 1UNC Neuroscience Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

Nature
|October 22, 2020
PubMed
まとめ

Cas9を用いた遺伝子治療は,アンジェルマン症候群 (AS) のマウスモデルにおいて,父親のUBE3A遺伝子を成功裏に再活性化させました. この早期の介入により 遺伝子の機能が回復し ASに関連する症状が改善され 病気を修正する治療法が提供されました

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